There were plenty of interesting developments in the gene editing and gene therapy space at the J.P. Morgan Healthcare Conference this year. Here are some top highlights.
Editas announced they will have the results from the Phase 1/2 clinical trial for EDIT-101 by the end of 2021. Editas made history in 2020 with EDIT-101, as it is the first CRISPR-based medicine in history to be directly administered to a patient. Most approaches collect cells from a patient, treat them outside the body, and then reintroduce the treated cells. Editas is also developing gene-editing therapies to treat cells outside the body. As announced they will initiate a Phase 1/2 clinical trial for EDIT-301 for the treatment of sickle cell disease.
“2020 was a momentous year towards our goal of developing differentiated, transformational medicines for people living with serious diseases. We began the year by making history with the first-ever clinical trial of an in vivo Gene EDITed medicine and ended the year by filing an IND for our first ex vivo cell medicine, EDIT-301,” said Cynthia Collins, President and CEO of Editas. “Building on these successes, I am confident our strong momentum will continue in 2021 as we dose additional patients and share clinical data from the BRILLIANCE trial, bring EDIT-301 into the clinic, file our third IND, and advance our in vivo and ex vivo Gene EDITed medicine programs.”
PTC Therapeutics is known for their success with protein restoration therapies such as Translarna and small-molecule splicing modifies such as PTC518, which is currently Phase 1 clinical trials. However, they announced that in 2021, they will launch their first potential gene therapy for the treatment of Aromatic l-amino acid decarboxylase (AADC) deficiency, a rare genetic disease that affects the brain. This therapy, PTC-AADC, will consist of a one-time dose of an adenovirus vector to introduce a functioning gene of the AADC gene. They will submit the Biologics License Application to the FDA in the first half of the year.
One of the most promising updates came for Verve Therapeutics, which announced that their therapy VERVE-101 caused a sustained reduction in blood low-density lipoprotein cholesterol, aka bad cholesterol. These results are from animal models, they looked at the liver of non-human primates after treatment with VERVE-101 and saw a 61% reduction in bad cholesterol. This was achieved with a single intravenous injection of VERVE-101, which is an adenine base editor messenger RNA and an optimized guide RNA targeting the PCSK9 gene.
“Coronary heart disease, the leading cause of death worldwide, is driven by cumulative exposure to LDL-C, and yet, current treatment approaches fail to sufficiently and durably reduce that exposure. As a result, many patients, particularly those with HeFH, suffer more heart attacks,” said Andrew Bellinger, M.D., Ph.D., chief scientific officer of Verve. “These exciting results show, for the first time, the durable and sustained effects of a single dose of our gene-editing medicine, validating this approach as a potential long-term, once-and-done treatment for coronary heart disease. Based on these and other preclinical data, we have initiated IND-enabling studies for VERVE-101 and are on track to begin clinical development in 2022.”
Another promising advancement for gene therapy came from Generation Bio. Although they are still in pre-clinical development for all of their therapies, they differentiate themselves by developing an alternative method to deliver their treatments. Currently, adeno-associated viruses (AAV) are used as a delivery method for a lot of gene-editing treatments; however, people can have a natural immunity to the viral vector, or they can cause an immune reaction. To address these, Generation Bio developed cell-targeted lipid nanoparticles, which in animal models do not trigger an immune response, and there is no natural immunity to them. More importantly, they developed a method to scale up the production of their cell-targeted lipid nanoparticles. If successful, this method could overcome some of the shortcomings faced by the adeno-associated virus.
By Daniel Ojeda, Ph.D.
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